Evidence map›Paper›PMID 20071242›Full record

ReviewClinical immunology (Orlando, Fla.)2010

Update on gene therapy for immunodeficiencies.

Donald B Kohn

Open access · greenAbstract readReview
In one paragraph

Review in Clinical immunology (Orlando, Fla.), 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed
8.0field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

22 citing papers in PubMed, 66 citations in OpenAlex.

  1. Gene therapy then and now: A look back at changes in the field over the past 25 years.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  2. CRISPR/Cas9-Directed Gene Trap Constitutes a Selection System for CorrectedInternational journal of molecular sciences · 2022
    Article
  3. Article
  4. Review
  5. Retroviral DNA Integration.Chemical reviews · 2016
    Review
  6. Review
  7. Article
  8. Bruton's tyrosine kinase: from X-linked agammaglobulinemia toward targeted therapy for B-cell malignancies.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2014
    Article
  9. Review
  10. Article
  11. Article
  12. Article
  13. Review
  14. Article
  15. Article
  16. Article
  17. Review
  18. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

1 author at 1 institution in 1 country.

Donald B KohnDepartments of Microbiology, Immunology and Immunology and Pediatrics, University of California, Los Angeles, 290D Biomedical Sciences Research Building, 615 Charles E. Young Drive South, Los Angeles, CA 90095, USA. dkohn@mednet.ucla.edu
University of California, Los Angeles · US

Funding

Transduction of Human Stem and Progenitor CellsP01HL073104 · NHLBI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI KOHN, DONALD B · 2003 to 2013
$18.3M
NHLBI NIH HHS P01 HL073104
6 · The paper itself

Abstract

Primary immune deficiencies (PID) are due to blood cell defects and can be treated with transplantation of normal hematopoietic stem cells (HSC) from another person (allogeneic). Gene therapy in which a patient's autologous HSC are genetically corrected represents an alternative treatment for patients with PID, which could avoid the immunologic risks of allogeneic HSCT and confer similar benefits. Recent clinical trials using gene therapy have led to immune restoration in patients with X-linked severe combined immune deficiency (XSCID), adenosine deaminase (ADA)-deficient SCID and chronic granulomatous disease (CGD). However, severe complications arose in several of the patients in whom the integrated retroviral vectors led to leukoproliferative disorders. New approaches using safer integrating vectors or direct correction of the defective gene underlying the PID are being developed and may lead to safer and effective gene therapy for PID.

Indexed as

Hematopoietic Stem Cell TransplantationClinical Trials as TopicGenetic TherapyGenetic VectorsHumansImmunologic Deficiency SyndromesRetroviridae

Identifiers

PMID20071242
PMCPMC2856741
OpenAlexW2030040544

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.